In-utero rescue of neurological dysfunction in a mouse model of Wiedemann-Steiner syndrome
Tinna Reynisdottir, Kimberley Jade Anderson, Andrew Brinn, Katheryn P. Franklin, Juan Ouyang, Ásbjörg Ósk Snorradóttir, Cathleen M. Lutz, Aamir R. Zuberi, Valerie B. DeLeon, Hans Tomas Bjornsson
This study demonstrates that in-utero restoration of KMT2A in a mouse model of WDSTS rescues neurological phenotypes, suggesting a therapeutic window during mid-gestation.
bioRxiv · Jul 19, 2024 · PreprintGenetics